Growth hormone deficiency in children MedDRA version: 20.0 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Cohort I: • Pre-pubertal children - Boys: Tanner stage 1 for pubic hair and testis volume 9.0 years and = 17.0 years at screening. - Boys: > 10.0 years and = 17.0 years at screening. - Confirmed diagnosis of GHD: a) for GH treatment naïve subjects, confirmed diagnosis within 12 months prior to screening as determined by two different GH stimulation tests, defined as a peak GH level of = 7.0 ng/ml. For children with three or more pituitary hormone deficiencies only one GH stimulation test is needed. b) for non-GH treatment naïve subjects, confirmed GHD diagnosis by investigator according to local practice - For GH treatment naïve subjects, no prior exposure to GH therapy and/or IGF-I treatment. - Open epiphyses; defined as bone age =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Cohort I, II and III: • Any clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing/length measurements: - Chromosomal aneuploidy and significant gene mutations causing medical "syndromes" with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors. - Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome, skeletal dysplasias. - Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. • Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age). • Concomitant administration of other treatments that may have an effect on growth, including but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD). • Prior history or presence of malignancy and/or intracranial tumour.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Cohort I: To evaluate the efficacy of multiple dose regimens of once-weekly NNC0195-0092 after 26 weeks of treatment in GH treatment naïve prepubertal children with GHD compared to once-daily hGH administration (Norditropin® FlexPro®) Cohort II and III: To evaluate the safety of once-weekly NNC0195-0092 during up to 208 weeks of treatment in children with GHD.;Secondary Objective: Cohort I: • To evaluate the safety of multiple dose regimens of once-weekly NNC0195-0092 during 26 weeks of treatment in GH treatment naïve prepubertal children with GHD. • To evaluate the efficacy and safety of multiple dose regimens of onceweekly NNC0195- 0092 for up to 364 weeks of treatment in GH treatment naïve pre-pubertal children with GHD compared to Norditropin® FlexPro®. • To investigate the impact of NNC0195-0092 relative to Norditropin® FlexPro® on wellbeing, psychosocial functioning and treatment satisfaction in GH treatment naïve prepubertal children with GHD. • To monitor NNC0195-0092 and Norditropin® PK throughout the trial.;Primary end point(s): Cohort I: Height velocity (HV) (cm/year) during first 26 week of treatment, measured as standing height with stadiometer Cohort II and III: Incidence of adverse events, including injection site reactions in children with GHD.;Timepoint(s) of evaluation of this end point: Cohort I: At baseline and after 26 weeks Cohort II and III: During at least 13 weeks and up to 208 weeks of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Cohort I: Efficacy Changes in the following variables will be used to address the primary objective: 1. Height standard deviation score (SDS) 2. HV SDS Safety The following endpoints will be used to support the secondary objectives of evaluation of safety: 3. Incidence of adverse events, including injection site reactions 4. Occurrence of anti-NNC0195-0092 and anti-hGH antibodies;Timepoint(s) of evaluation of this end point: 1. + 2.: From baseline to end of main trial period (week 26) 3. + 4.: up to 364 weeks of treatment | — |
Countries
Austria, Belgium, Brazil, European Union, Germany, India, Israel, Japan, Slovenia, Sweden, Turkey, Ukraine, United States
Contacts
Public ContactClinical Transparency (2834)
Novo Nordisk A/S
Outcome results
None listed